Metastatic Cholangiocarcinoma: Targeted Therapy and NGS


Rachna Shroff, MD: The introduction of targeted therapy for cholangiocarcinoma is absolutely an exciting area right now because it has led to rapid drug development and new therapeutic options for our patients. I think what’s exciting about it is that we are learning that one size does not fit all for cholangiocarcinoma. By molecularly profiling these patients, we are able to find potentially actionable targets for which we have drugs. That’s basically what I tell my patients, that this is not just the era of chemotherapy anymore. We have novel drugs and novel targets that we can go after effectively.

Next-generation sequencing in treating metastatic cholangiocarcinoma should be one of the first things that we do in a newly diagnosed patient or the first time we meet a patient. I cannot explain or emphasize that enough to patients, to oncologists, and other providers because, as I said, it is an additional area of therapeutic options that we could be missing out on if we don’t do next-generation sequencing. I describe it to patients as understanding the mutations that they acquire over the course of their life that could have led to the growth and progression of cholangiocarcinoma.

The only way to truly understand the biology and what targets we can go after is by using next-generation sequencing. The number I usually quote for intrahepatic cholangiocarcinoma is about 30% to 40% of patients for whom we find potentially actionable targets. That’s a really large number in terms of therapeutic options. Even though it’s a little bit smaller for extrahepatic cholangiocarcinoma, it is still a relevant thing to be doing in these patients as well.

Molecular testing should be ordered, in my opinion, at the time of diagnosis and that is for 2 different reasons. Number 1) There are now clinical trials for patients with newly diagnosed metastatic cholangiocarcinoma that could be options if we find and identify specific alterations. Also, because it unfortunately takes some time to get that next-generation sequencing result back, and that lag happens from the time in which it takes the tissue to get to whatever platform we’re using in terms of a company for sequencing, as well as the actual time it takes once they get the tissue in hand for doing the full profiling. I always tell my patients I order it in parallel as I’m working them up from the time of diagnosis. There’s also some debate that there is a role for retesting or reprofiling patients even after subsequent lines of therapy. If I meet a patient who has not had next-generation sequencing, it doesn’t matter where they are in their cancer journey. I absolutely order it at that time.

Transcript edited for clarity.

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